Lucia Rugeri1, Roseline d'Oiron2, Annie Harroche3, Valérie Proulle4, Guillaume Mourey5, Emmanuelle De Raucourt6, Dominique Desprez7, Nathalie Itzhar Baikian8, Brigitte Pan Petesch9, Annie Borel-Derlon10, Sophie Combe11, Birgit Frotscher12, Abel Hassoun13, Hasan Catovic14, Diane Bracquart14, Marc Trossaërt15. 1. Unité Hémostase Clinique, Hôpital Louis Pradel, Hospices Civils de Lyon, Bron, France. 2. Centre de Référence pour le Traitement des Maladies Hémorragiques, Hôpital Bicêtre, Paris, France. 3. Centre de Traitement de l'Hémophilie, Hôpital Universitaire Necker Enfants Malades, Paris, France. 4. Service Hématologie Biologique, Hôpital Bicêtre, AP-HP, Paris, France. 5. Centre de Référence et de Traitement de l'Hémophilie, Etablissement Français du Sang, Besançon, France. 6. Service d'Hématologie Biologique, Hôpital Beaujon, Paris. 7. Centre de Traitement de l'Hémophilie, CHU Strasbourg, Strasbourg, France. 8. Service d'Hématologie Biologique, Hôpital Lariboisière, Paris, France. 9. Unité Hémostase Service Hématologie, CHU Brest, Brest, France. 10. Centre Régional de Traitement des Maladies Hémorragiques, Institut de Biologie Clinique, Hôpital Charles Nicolle, Rouen, France. 11. Service Hématologie Biologique, Hôpital Bicêtre, Le Kremlin-Bicêtre, Paris. 12. Service d'Hématologie Biologique, CHU, Nancy, France. 13. Centre de Traitement de l'Hémophilie, Hôpital Simone Veil d'Eaubonne, Montmorency. 14. CSL Behring, Paris, France. 15. Centre de Traitement de l'Hémophilie, CHU Hôtel-Dieu Nantes, Nantes, France.
Abstract
BACKGROUND: In patients with moderate to severe qualitative and quantitative von Willebrand disease (VWD), even minor surgical procedures can be associated with a risk of life-threatening bleeding. Treatment strategies vary according to the levels of von Willebrand factor (VWF) and Factor VIII (FVIII). The aim of this study was to evaluate the effectiveness and the safety of Voncento® (CSL Behring, Marburg, Germany), a plasma-derived FVIII/VWF concentrate (ratio 1:2.4), during surgeries performed in patients with inherited VWD. MATERIALS AND METHODS: The OPALE study, a French multicentre observational study, was carried out from May 2016 to May 2019. It evaluated and analysed patients with inherited VWD (any type) requiring treatment with Voncento® who underwent surgery. RESULTS: In total, 92 patients were enrolled, and 66 patients underwent 100 surgical procedures: 69 minor and 31 major surgeries conducted in 30 patients with type 1, 50 patients with type 2, and 20 patients with type 3 VWD. During minor surgeries, the median number of infusions was one (range: 1-9), the pre-operative loading dose was 41 IU VWF:RCo kg-1 (range: 18-147), and the total dose was 63 (range: 18-594). During major surgeries, the number of infusions was 4 (range: 1-23), the pre-operative loading dose was 43 (range: 25-66) IU VWF: RCo kg-1, and the total dose was 155 (range: 40-575). The median FVIII:C levels ranged from 78 to 165 IU dL-1 during 5 days after minor surgeries and from 86 and 167 IU dL-1 during 11 days after major surgeries. VW:RCo levels ranged between 35 and 65 IU dL-1 and between 34 and 76 IU dL-1 after minor and major surgeries, respectively. The overall clinical effectiveness was qualified as "excellent" or "good" in 99% of patients. No thrombotic events related to Voncento® were recorded. DISCUSSION: The present study suggests that Voncento® is an effective and well-tolerated therapy for the peri-operative management of patients with all VWD types.
BACKGROUND: In patients with moderate to severe qualitative and quantitative von Willebrand disease (VWD), even minor surgical procedures can be associated with a risk of life-threatening bleeding. Treatment strategies vary according to the levels of von Willebrand factor (VWF) and Factor VIII (FVIII). The aim of this study was to evaluate the effectiveness and the safety of Voncento® (CSL Behring, Marburg, Germany), a plasma-derived FVIII/VWF concentrate (ratio 1:2.4), during surgeries performed in patients with inherited VWD. MATERIALS AND METHODS: The OPALE study, a French multicentre observational study, was carried out from May 2016 to May 2019. It evaluated and analysed patients with inherited VWD (any type) requiring treatment with Voncento® who underwent surgery. RESULTS: In total, 92 patients were enrolled, and 66 patients underwent 100 surgical procedures: 69 minor and 31 major surgeries conducted in 30 patients with type 1, 50 patients with type 2, and 20 patients with type 3 VWD. During minor surgeries, the median number of infusions was one (range: 1-9), the pre-operative loading dose was 41 IU VWF:RCo kg-1 (range: 18-147), and the total dose was 63 (range: 18-594). During major surgeries, the number of infusions was 4 (range: 1-23), the pre-operative loading dose was 43 (range: 25-66) IU VWF: RCo kg-1, and the total dose was 155 (range: 40-575). The median FVIII:C levels ranged from 78 to 165 IU dL-1 during 5 days after minor surgeries and from 86 and 167 IU dL-1 during 11 days after major surgeries. VW:RCo levels ranged between 35 and 65 IU dL-1 and between 34 and 76 IU dL-1 after minor and major surgeries, respectively. The overall clinical effectiveness was qualified as "excellent" or "good" in 99% of patients. No thrombotic events related to Voncento® were recorded. DISCUSSION: The present study suggests that Voncento® is an effective and well-tolerated therapy for the peri-operative management of patients with all VWD types.
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